International Expert Team Meets to Finalize First-Ever DM Care Recommendations for Doctors

As many MDF community members know, the foundation has been working with a group of expert clinicians from across Western Europe, the U.K, Canada and the U.S. for the past year to develop consensus-based clinical care recommendations for myotonic dystrophy. We first talked about this project as part of a larger story about our three year, multi-million dollar drug development and care expansion effort. We have made excellent progress since we first mentioned this program in May 2015, and we are pleased to bring you an update on this unique initiative.

What Are Standards of Care and Why Are They Important?

Standards of care are critically important to help ensure that all patients with a particular disease receive appropriate and beneficial clinical care. They are typically based on rigorous data captured through large, anonymous research studies, and are usually developed and issued by the leading professional organization for that particular disease. In the case of myotonic dystrophy, that organization in the U.S. is the American Academy of Neurology (AAN). AAN has been working on an Evidence-Based Guideline for myotonic dystrophy for over five years, and hopes to publish it in 2017.

Clinical care guidelines will be particularly meaningful for the DM community because, as our community knows well, many doctors who do not see DM patients regularly are not aware of the range and variation in symptoms that are part of living with myotonic dystrophy. They do not necessarily know what testing, exams and follow up are appropriate for people living with DM, and they may miss important impacts to some organ systems. Often it is the DM patient who educates the physician about the disease and the care that is needed. MDF is committed to helping ensure that care recommendations are available and delivered to the clinical professionals who treat DM patients.

The DM Guideline, when it comes out, will be published and disseminated to neurologists in the U.S. It will not address most of the organ systems and symptoms of importance to myotonic dystrophy patients because the studies needed to gather the evidence required to establish a guideline have not been conducted.

MDF is working with the AAN to support the eventual dissemination and publication of the DM Evidence-Based Guideline when it is ready, and most of the members of our Scientific Advisory Committee are part of the team involved in its creation. Dr. Tetsuo Ashizawa, formerly the head of neurology at the The University of Florida and now leading neurology research at Houston Methodist, is the lead clinician on The DM Guideline.

What are Care Considerations and How Are They Different?

MDF has had a number of meetings with the U.S. Centers for Disease Control (CDC) and the AAN to understand what the newest publication date is for the evidence-based guideline, and how robust the final publication will be. The AAN and CDC have heavily encouraged MDF to pursue the development of consensus-based care recommendations in order to ensure that comprehensive care recommendations are available to all clinicians treating DM patients because a comprehensive evidence-based guideline is many years away.

Consensus-driven care recommendations are clinical care recommendations developed by experts in the appropriate disease field, who work through a formal consensus-development process to come to agreement on how patients across many countries and continents should be treated to help patients maintain health and improve their quality of life.

The Myotonic Dystrophy Care Considerations

In the case of the DM Care Considerations, MDF has assembled a truly world-class team of 60 expert clinicians from across the globe, in fields as diverse as cardiology, pulmonology, occupational therapy, pain and family planning, to work through a consensus-building process to create the first-ever clinical care recommendations for adults with myotonic dystrophy type 1. Care Considerations for congenital myotonic dystrophy and myotonic dystrophy type 2 will follow very shortly. Both the CDC and AAN are partners on the MDF Care Considerations project and are providing significant support to the project. MDF has also teamed up with Treat-NMD, Muscular Dystrophy UK and organizations in Canada and Western Europe to ensure that these guidelines are appropriate and targeted to patients in many different countries and care situations.

What’s Been Accomplished?

The project formally kicked off at the 2015 MDF Annual Conference, where MDF held a meeting with the international working group to provide an overview of the project scope, the consensus-development methodology, and study area assignments. Eight multi-system study areas were established, with committee chairs and working groups for each. Click here to see the full international list of Care Considerations study areas and Working Group participants.

MDF created and circulated to the working group a draft list of care recommendations developed from the MDF Toolkit and a short list of key published research. The expert clinicians then met via conference call and email, reviewing and editing the recommendations for their study areas over the course of the winter. MDF assembled an updated draft of care considerations with the edits included, and then brought all available working group members to Miami to discuss the revised document and work toward final recommendations and consensus. The meeting was a significant success, and MDF is now assembling the final draft document to circulate for a last round of reviews to the working group.

What’s Next?

The next steps for the DM Care Considerations project will be extensive and will involve multiple international partner organizations. Once the working group has agreed on the final list of care recommendations, MDF will develop executive summary and full versions of the document, as well as a draft publication for submission to a neurology journal. The full version will be submitted to professional associations of clinical professionals for open comment and to accreditation organizations in Western Europe, the U.K., Canada and the U.S. (AAN) to begin the final approval and dissemination process. Additional international organizations will be significant partners in distributing the final recommendations to care providers internationally.

MDF will also pursue an insurance policy document to help convince insurance companies in the U.S. to provide reimbursement for these care recommendations, and MDF has developed a draft update policy to ensure that these recommendations stay current. Finally, MDF will work with the working group and our international partners to develop and implement an assessment program to measure whether these recommendations are being used and are improving care for DM patients.

We plan to have final approved documents ready by the end of 2016. As always, MDF will keep you posted on this and our other research and care projects as we move forward. Questions? Contact MDF via email or phone at 415-800-7777.